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Transformative Therapies from Bench to BedsideCorporate PresentationMarch 2014
Slide 2
Forward Looking Statements
This presentation contains forward‐looking statements and information that are based on the beliefs of the management of Capricor Therapeutics, Inc. (Capricor) as well as assumptions made by and information currently available to Capricor. All statements other than statements of historical fact included in this presentation are forward‐looking statements, including but not limited to statements identified by the words “anticipates,” “believes,” “estimates,” and “expects” and similar expressions. These statements reflect Capricor’s current views with respect to future events, based on what we believe are reasonable assumptions; however, the statements are subject to a number of risks, uncertainties and assumptions. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward‐looking statements. More information about these and other risks that may impact our business are set forth in the Form 10‐K of Nile Therapeutics, Inc. (Nile) for the year ended December 31, 2012, as filed with the Securities and Exchange Commission on June 21, 2013, in Nile’s Quarterly Report on Form 10‐Q for the quarter ended June 30, 2013, as filed with the Securities and Exchange Commission on August 14, 2013, in Nile’s Definitive Proxy Statement on Schedule 14A, as filed with the Securities and Exchange Commission on October 10, 2013, in Nile’s Quarterly Report on Form 10‐Q for the quarter ended September 30, 2013, as filed with the Securities and Exchange Commission on November 14, 2013, and in Form 8‐K/A, as filed with the Securities and Exchange Commission on January 24, 2014. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those in the forward‐looking statements. Further, Capricor’s management does not intend to update these forward‐looking statements and information after the date of this presentation.
Slide 3
Capricor Investment HighlightsCAPR is a publicly‐traded, biotech company focused on developing novel therapeutics to prevent and
treat heart disease Strategically positioned to treat heart failure
o 2005: Capricor, Inc. was founded to develop autologous cardiac derived cell therapy
o November 2013: Capricor Therapeutics was created through merger of Capricor, Inc. with Nile Therapeutics, Inc.
Capricor, Inc. ‐ capitalized through private and public fundingo Equity investments of $12M
o Grants of $7M
o CIRM Loan of $19.8M
o Janssen collaboration income of $12.5M
o Total of $39.5M of non‐dilutive capital Developmental pipeline with CAP‐1002 in an ongoing clinical trial
o Cardiosphere‐derived cells (CDCs): CAP‐1001 and CAP‐1002
o Cenderitide and CU‐NP
o Regenerative medicine product in pre‐clinical development
Exclusive licenses with top academic and research institutions
Slide 4
Source of Cash Amount CommittedTiming of Funding Use of Cash
NIH SBIR Grant $945K Yes Complete R&D
NIH Grant $3M Yes Complete Phase I Portion of ALLSTAR
CIRM Loan Award
$19.8M Yes Ongoing Phase II Portion of ALLSTAR
NIH SBIR Grant $3M Yes 2014 CDC Clinical Trial
Janssen $12.5M Yes Obtained New Product Development and Manufacturing
TOTAL: $39.5M of Non‐Dilutive Capital
Capricor: Capital Efficient and Operationally Leveraged
Slide 5
Main Product Candidates CAP‐1002
o Capricor’s lead product candidateo Allogeneic, off‐the‐shelf CDC producto ALLSTAR Phase I/II Trial
o Evaluated in ALLSTAR Phase I trial, completeo Currently enrolling patients in ALLSTAR Phase II trial, ongoing
o Entered into collaboration and exclusive license option with Janssen Biotech, Inc.o DYNAMIC (CSMC sponsored )Phase I trial evaluating CDCs, likely CAP‐1002
Cenderitideo Belongs to class of drugs called natriuretic peptideso Designed by Mayo Clinic scientists to be only dual NP receptor agonist, differentiated
MOAo Potential to be superior HF treatment solution by offering several therapeutic benefitso Being developed as an outpatient therapy for patients to be taken for 90 days following
admission for ADHF, the "post‐acute" period
Slide 6
Janssen Deal HighlightsIn December 2013, Capricor entered into collaboration and exclusive license option with Janssen Biotech, Inc.
Capricor received an upfront payment of $12.5M
Capricor and Janssen to collaborate on elements of cell manufacturing
Janssen has option to enter into exclusive license agreement for CAP‐1002 up to 60 days after receipt of six month ALLSTAR Phase II data o If exercises option, Capricor to receive an upfront license fee and additional
milestone payments which may total up to $325M
Double‐digit royalty to be paid on commercial sales of licensed products
Important validation of lead product, CAP‐1002, and underlying science
Supports the continued development of CAP‐1002
Slide 7
Capricor Therapeutics Pipeline
IND Received Enrollment Enrollment
Complete
Data Analysis and Publication
ALLSTAR – Phase I(14 patients, funded in part by NIH)
CADUCEUS – Phase I (CSMC and JHU sponsored)
DYNAMIC – Phase I(CSMC sponsored)
ALLSTAR – Phase II(300 patients, funded in part by CIRM)
Cenderitide
Slide 8
CADUCEUS: Phase I Trial of CAP‐1001
Study Overview Prospective, randomized Phase I trial Evaluated autologous CDCs, CAP‐1001 Patients with reduced ejection fraction
(EF) following MIo 25 patients total (17 CDCs; 8 SOC)
Delivery: Standard interventional catheters to infarct‐related artery
Sponsored by Cedars‐Sinai and JHU Results published in the Lancet
(Makkar, 2012)
Study Results Potential for therapeutic regeneration
in cardiac tissueo Reduced scar size and increased healthy
heart muscleo Significant reduction in infarct size (IS)
Proof‐of‐concept study demonstrating CDC therapy reduced scar size and increased healthy heart muscle
Slide 9
The CADUCEUS Study: CDCs and Infarct Size
Both groups (CDC and CTRL) started with IS of 24%
Patients treated with CDCs saw a reduction of IS to 12.5%
This moved patients from a highrisk group to a low risk group.
CTRL patients saw no change in IS, which puts them at great risk for adverse events
CDCs
Wu, E. et al. Heart 94, 730‐736 (2008).
Slide 10
Limitations of Autologous TherapyRestrictions of effective autologous stem cells transplantation, in which cells are removed, stored, and returned to same patient
Timing constraints o 3‐5 weeks to obtain and then develop CDCs
Higher Expense
Significant Risko Harvesting procedureo Manufacturing failure
Suboptimal QA/QC
Inter‐patient variability in cell potency
Slide 11
Advantages of Allogeneic CDCs
Donor Heart
Modified from Smith et al., Circulation 2007
Several advantages to allogeneic stem cell transplantation in which patient receives cells from a healthy donated heart Donors pre‐screened by organ procurement organizations Semi‐automated explant creation Large master cell banks created from each donor Batch production of cryopreserved doses FDA approved for Phase II clinical trial use
Explant Culture Cardiosphere Formation CDC Expansion Patient
Dose
CAP‐1002
Slide 12
ALLSTAR: Phase I/II Trial of CAP‐1002
Study Overview Evaluating safety and efficacy of off‐the‐
shelf allogeneic CDCs, CAP‐1002 Ejection Fraction (EF): EF ≤ 45% and IS ≥
15% (MRI) Dual cohort (early and late post MI)
o Phase I: Open‐label cohort o Phase II: Double‐blinded, randomized,
placebo‐controlled (currently enrolling)
U.S. multi‐site trial: 30‐40 sites projected Delivery: Intracoronary artery
ALLogeneic heart STem cells to Achieve myocardial Regeneration
Study Highlights Phase I: (n=14) 1 month safety
endpoint o Enrollment completeo Cleared to proceed to Phase II
Phase II: (n=300) 6‐month efficacy data, est. 2016
o LV Structure and Remodeling o LV global functiono Pt. Symptomatic Statuso Biomarkers
Slide 13
ALLSTAR: Trial Design
DSMB Review
Safety Cohort (N=14)
CAP -1002 (N=87)
Placebo (N=43)
CAP -1002 (N=87)
Placebo (N=43)
CAP -1002 (N=7)
CAP -1002 (N=7)
Phase 1 Phase 2
Randomized Cohort (N=260)
CAP -1002 (N=9)
CAP -1002 (N=5)
FDA Approved
* Funded by NIH ARRA RC3 * Funded by CIRM DT2
Slide 14
ALLSTAR: Phase I Primary Endpoint
ALLSTAR Phase I met the primary safety endpoint
1°Safety Endpoint: 1 month post‐infusion o NO Acute Myocarditis attributable to CAP‐1002o NO Death due to VT/VFo NO Sudden Deatho NO Major Adverse Cardiac Events (MACE)
NIH DSMB approved advancement to Phase II
Slide 15
ALLSTAR: Phase I Immunology
Antibody response monitoring (DSA) revealed low level antibodies (MFI <5000) o 4 subjects with pre‐existing DSAs: 1 resolved, 3 persistedo 4 subjects with de novo DSAs: 2 resolved, 2 pending
follow up
No clinical sequelae seen (acute myocarditis, LV dysfunction)
Cellular response monitoring (ELISPOT) revealed no de novo responses
* All data reflect follow up thru Jan 2014
Slide 16
ALLSTAR: Phase II Endpoints
1°Safety Endpoint: 1 month post‐infusion o Acute Myocarditis attributable to CAP‐1002o Death due to VT/VFo Sudden Deatho MACE
1°Efficacy Endpoint: 12 months post‐infusiono Relative improvement of Infarct Sizeo Strata will be evaluated separately o Power >80% to detect a 15% difference between
CAP‐1002 and placebo
Slide 17
DYNAMIC: Phase Ia Trial of CDCs
Study Overview IND Sponsor: Cedars‐Sinai Medical Center (E. Marbán)
Patient criteriao Ischemic or non‐ischemic DCM with LVEF ≤ 35%o NYHA Class III or ambulatory Class IV heart failureo Hospitalization for heart failure within 12 months
Delivery: Non‐occlusive intracoronary infusion in 2‐3 vesselso Phase Ia: open‐label (N=14, single center): safety
Dilated cardiomYopathy iNtervention with Allogeneic MyocardIally‐regenerative Cells
Slide 18
Well Positioned for Success in Cell Therapy
CDCs’ Unique Mechanism of
Action
CDCs demonstrated a reduction in infarct size and cardiac regeneration in the POC CADUCEUS trial
Ease of Use Allogeneic cells allow for an “off‐the‐shelf” product
Stable Supply Chain
Donor hearts provide a sustainable source of tissue from which CDCs can be derived
Scalable Manufacturing
One heart can provide thousands of doses, with a targeted gross margin of over 90%
Novel Technology IP portfolio
Experienced Leadership World renown scientific and management teams
Slide 19
Senior Management & Board of Directors
Senior Management
Chief Executive Officer Linda Marbán, Ph.D.
EVP & General Counsel Karen Krasney, J.D.
VP of Medical Affairs Andrew Hamer, M.D.
VP of Research & Development Rachel Smith, Ph.D.
VP of Finance AJ Bergmann, M.B.A.
Board of Directors
Executive Chairman Frank Litvack, M.D.
Linda Marbán, Ph.D.
Dave Musket
Earl M. (Duke) Collier, Jr.
George W. Dunbar, Jr.
Louis Manzo
Louis J. Grasmick
Joshua Kazam
Gregory Schafer